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Published on Wednesday, January 31, 2018

First John W. Walsh Translational Research Award granted

MIAMI/BOSTON, January 31, 2018 – The Alpha-1 Foundation celebrates the first granting of the John W. Walsh Translational Research Award in Alpha-1 Antitrypsin Deficiency, an honorable award named after John W. Walsh, co-founder of the Alpha-1 Foundation, a visionary man who dedicated his life’s work to finding a cure for Alpha-1 Antitrypsin Deficiency and helping those affected by Alpha-1.

The peer reviewing Committee received four very strong submissions, and the award was granted to Joseph Kaserman, MD, instructor of medicine at Boston University School of Medicine (BUSM) and a physician in Pulmonary, Allergy, Sleep and Critical Care Medicine at Boston Medical Center.

Alpha-1 Antitrypsin Deficiency (Alpha-1) is a genetic (inherited) condition – it is passed from parents to their children through their genes. Alpha-1 may result in serious lung disease in adults and/or liver disease at any age.

The inaugural John W. Walsh Translational Research Award will be used by Kaserman to address a long outstanding question in the field of whether Alpha-1 carriers (these are individuals who have one normal Alpha1 gene (M) and one mutated gene (Z) have an increased risk of developing liver disease. As up to 2% of the U.S. population could be an Alpha-1 carrier there remains a critical need to address this important question.

For his research Kaserman will be working with specialized adult stem cells named induced pluripotent stem cells (iPSCs), which are stem cells that have been created from patients with Alpha-1 Antitrypsin Deficiency. Importantly, iPSCs contain all the genetic information of the original patient, and provide the ability to compare cells from patients that have the same mutation but different disease severity, i.e. patients who have the Z-mutation with or without liver damage.

To understand the risk associated with having either one or two Z genes, Kaserman will take advantage of a technology known as CRISPR that allows for altering or “editing” the DNA of cells including iPSCs that he has made from Alpha-1 patients. Using this approach, Kaserman will be able to conduct experiments comparing “ZZ”, “MZ”, and “MM” cells that all come from the same individual and thus are genetically identical, varying only at the site of the Alpha-1 Antitrypsin gene that has been edited.

Kaserman will then create liver cells from these iPSCs and examine whether MZ cells accumulate more misfolded AAT protein or are more susceptible to injury than MM cells. As there remain no approved therapies for Alpha-1 liver disease this study will also test new therapeutic agents targeting the mutated Z-protein within cells to see if this will protect the susceptible liver cells from injury.

Overall this study has the potential to not only address the concerns of many Alpha-1 carriers about their risk of developing liver disease, but also to find new treatments for Alpha-1 related liver disease helping both Alpha-1 carriers as well as Alpha-1 patients.

The award consists of up to a total of $225,000 ($75,000/year) over three years. The Alpha-1 Foundation, the Center for Regenerative Medicine of Boston University and Boston Medical Center, congratulate Kaserman, and look forward to the research findings, which will surely bring us closer to finding the cure for Alpha-1 Antitrypsin Deficiency (Alpha-1).

Back in October 2016, in recognition of John Walsh’s many contributions, Mark Brantly, MD, announced a $225,000 gift from himself and his wife Judy Lew, MD, to the Foundation during the Alpha-1 Donor Recognition Dinner. The gift was intended to be the first to establish the John W. Walsh Translational Research Award.

Brantly, professor of medicine, chief of molecular genetics and microbiology and Alpha-1 Foundation research professor at the University of Florida, said about John Walsh that “Judy and I believe that the best way to honor and thank him is to help carry on his commitment to a cure through this research award.”

“This is a tangible, concrete way to honor John and continue the work he was so committed to do,” said Henry Moehring, President and CEO of the Alpha-1 Foundation. “The Alpha-1 Foundation is honored that AlphaNet was able to make a matching contribution to this grant, enhancing even further the value we give to John’s legacy and his commitment to research”, added Moehring.

John Walsh’s leadership and passion helped shape the Alpha-1 Foundation into a successful organization and he was responsible for many of the Foundation’s accomplishments over more than two decades.

“This award, aimed specifically at translating research findings into medical practice, is intended to carry on Walsh’s commitment to find a cure for Alpha-1 Antitrypsin Deficiency”, expressed Moehring.

“We are extremely gratified that Kaserman has received this award, both because it is a huge honor to be selected to help carry out John’s vision and because of the significance this kind of award has in sustaining the career of a young researcher,” said Andrew Wilson, MD, Associate Professor of Medicine at BUSM, and Director of The Alpha-1 Center at Boston University and Boston Medical Center.

“John Walsh had a major impact on my career and on the Alpha-1 research that we carry out at the Center for Regenerative Medicine of Boston University and Boston Medical Center. John spoke openly about hopes for a cure for AATD and encouraged us to pursue major rather than incremental advances in our research efforts. He served as a mentor both to me and to Dr. Kotton and was instrumental in bringing us into the Alpha-1 community,” added Wilson.

“I am both excited and extremely humbled to have been selected as the inaugural recipient for the John W. Walsh Translational Research Award. Although I never had the pleasure of meeting John personally, I have heard numerous anecdotes from my mentor Dr. Wilson about how committed John was to the Foundation and how relentless he was about finding a cure for Alpha-1. As a pulmonologist caring for patients suffering from the complications of Alpha-1, I am continually reminded that our current therapies remain grossly inadequate, but this only reinforces the clinical relevance of our research especially if we are going to one day realize John’s goal of finding a cure for Alpha-1,” said Kaserman.

Boston Medical Center and the Boston University Pulmonary Center have long-standing ties to the Alpha-1 community through Gordon Snider, MD, the first chief of pulmonary medicine at Boston University, and a seminal clinician, teacher and researcher in the field. Snider’s work demonstrating the role of neutrophil elastase in the pathogenesis of emphysema, and interest in the role of Alpha-1 Antitrypsin (AAT) in protecting the lung against it, created a legacy that lives on today in the form of the research that Kaserman and Wilson carry out in their lab, as well as in the Alpha-1 Center, where they see patients with AATD.

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About the Alpha-1 Foundation: The Alpha-1 Foundation is committed to finding a cure for Alpha-1 Antitrypsin Deficiency and to improving the lives of people affected by Alpha-1 worldwide. The Alpha-1 Foundation is a not-for-profit Florida corporation founded in 1995 by John Walsh, Sandy Lindsey and Susan Stanley, three people diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1). The Alpha-1 Foundation has invested more than $71 million to support Alpha-1 research and programs at 112 institutions in North America, Europe, the Middle East and Australia. For more information, visit www.alpha1.org.

About the Center for Regenerative Medicine (CReM): The Center for Regenerative Medicine (CReM) is a collaborative effort among scientists at Boston University and the Boston Medical Center that aims to advance stem cell research and regenerative medicine for the sake of patients. Drs. Kaserman and Wilson’s lab is in the CReM, a state-of-the-art, multidisciplinary stem cell research facility that is home to 7 “primary investigators” (PIs) and their respective trainees. Dr. Kotton is CReM director and Wilson is one of the CReM PIs. Since its founding in 2013, AATD has been a major focus of research at the CReM, which houses the world’s largest repository of iPSCs created from patients with AATD. In keeping with a dedication to the philosophy of “Open-Source Biology”, CReM iPSCs are shared free of charge with researchers around the world to advance AATD research. Wilson, Kaserman, and Kotton provide clinical care to AATD patients and families at the Alpha-1 Center. The Alpha-1 Center features clinicians from multiple specialties, all experienced in caring for Alphas, to address all aspects of AATD. For more information about the CReM and the Alpha-1 Center, visit www.bumc.bu.edu/stemcells and www.alpha-1center.org.

Translational research in the biomedical field is understood as the application of basic knowledge acquired in the research laboratory to clinical practice, to improve medical care. This type of research is a necessary element for the success of any strategy that aims to improve the health of citizens through specific programs.

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